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April 30, 2004
Election 2004 Update - Sort Of
A search through news articles for the last month shows that there has been virtually no discussion by the presidential candidates on health care. What discussion there has been could be phrased this way: "It needs to be cheaper."
Somehow I doubt that expanding medical research will become a major topic in this campaign. (I know a shocking prediction.)
Posted by Dave at 10:20 PM | Comments (0) | TrackBack
April 29, 2004
Brain Stem Cells Created From Bone Marrow
This is big news. But due to politics surrounding stem cell research won't get the play it deserves...
Researchers have found a way to create brain stem cells from bone marrow cells. These brain stem cells could someday be used to restore the brain from the damage caused by Huntington's Disease, or at least help protect it.
Why this is big news (a clip from the article)...
"Still, cells derived from a patient's own bone marrow would provide a way around certain medical and moral issues.
Tissue transplants sourced from other than the patient's own body can be rejected by the body's immune system, Saporta pointed out. Suppressing this immune response with powerful drugs "is very, very onerous therapy, it's really hard on the individual and the system," he said. "If you can find a way of minimizing the amount of immunosuppression you have to do -- or not have to do any at all --- that's the ideal situation."
Stem cells derived from bone marrow stromal cells also avoid moral and legal issues surrounding embryonic stem cells. "Bone marrow donations are done all the time, there are thousands done every day in the United States," Saporta said. "There's not the ethical issue here of obtaining stem cells from an embryo."
Caveats abound...read the full article for more details
Posted by Dave at 04:22 PM | Comments (0) | TrackBack
April 28, 2004
Gene Therapy Trial Shows Promise
It's for Alzheimers, but the technique is applicable to Huntington's Disease.
What they did:
"They took skin cells called fibroblasts from each patient, and used a genetically modified virus to infect them with the NGF gene. The cells were allowed to multiply, then were injected into the brain, in a region called the nucleus basalis, which is severely affected by the disease. "They act as biological pumps for the long term administration of NGF in the brain," says Tuszynski."
There's a lot of interesting detail in the article and you can read it here.
Posted by Dave at 04:39 PM | Comments (1) | TrackBack
April 27, 2004
Amarin Buys An Opinion
Amarin paid a company $25,000 to provide an investment opinion.
No, that isn't that unusual. The investment opinion comes from J.M. Dutton & Associates and this press release seems reasonably accurate. They consider Amarin to be a "Speculative Buy" which is Wall Street talk for "a gamble". This gave the stock a nice little "goose" as it had been trading slightly about the dreaded $1/share mark. The price declined in late trading but it was still up 6% for the day.
For those who don't know...Amarin has LAX-101 (a Huntington's Disease drug) entering a second round of Phase III drug trials. Here's the press release for your enjoyment.
Posted by Dave at 08:37 PM | Comments (0) | TrackBack
April 26, 2004
WP Backs Genetic Discrimination Bill
The influential Washington Post has published a house editorial backing the passage of the Anti-discrimination bill that is currently stalled in a House subcommittee. Here is a key point they make (which has been made here also):
"But this promise is tempered by the risk that individuals' genetic heritage could be used against them. Employers could refuse to hire, or could fire, those predisposed to develop a disease. An insurer could refuse coverage, or charge higher premiums, on the basis of genetic tendency. And the specter of such discrimination could deter people from undergoing genetic testing, thus potentially undermining their own care and slowing the pace of scientific discovery."
Posted by Dave at 11:47 PM | Comments (0) | TrackBack
April 25, 2004
Medicare Chronic Care
There's a new health care blog "Healthsignals New York" (found via the Health Care Blog) on the federal government's new chronic care improvement program. You can get the details here.
Posted by Dave at 10:00 PM | Comments (0) | TrackBack
April 24, 2004
Another "It's A Bird..." Review
Steven T. Seagle gets another review of his graphic novel "It's a bird..." - this time from Newsday. Not as long as the last one, but still interesting. Here's an excerpt:
"Steven, the author's alter ego, seems almost repelled by this opportunity of a comic book writer's lifetime. He has issues - or "back issues," as he puts it - with Superman dating to his childhood, when his grandmother died from Huntington's disease. The disease's hereditary path casts a pall on both Steven and his father, who goes missing as Steven's relationship with his girlfriend crumbles."
Posted by Dave at 11:33 PM | Comments (0) | TrackBack
April 23, 2004
Convicted!
The jury didn't waste much time in convicting Queen Elnora Graham for stealing over $47,000 from an elderly couple - one with Huntington's Disease. Deliberations took less than three hours.
If we're lucky, she'll serve the maximum 15 years in jail. Most likely she'll serve much less. The jury didn't believe Graham when she said she was buying lingerie and leather miniskirts for an elderly, invalid woman in a wheelchair.
As I said yesterday, she should have plead guilty.
A representative from the South Florida HSDA chapter sent a thank you to Naples Daily News for covering this story. If you would like to do the same you sent a thank you to cwcolby (at) naplesnews (dot) com.
Posted by Dave at 08:21 AM | Comments (0) | TrackBack
April 22, 2004
Sounds Like She's Guilty
There's a woman on trial in Naples, Florida who is accused of stealing from a woman with Huntington's Disease and her husband a stroke victim.
The woman on trial, Queen Elnora Graham, was working for Bruce and Betty Stromgren as a home health aide as they were both in wheelchairs. Graham also did a little shopping - to the tune of over $47,000 in cash and merchandise.
Graham, (are you sitting down for this?) states she was buying for Betty. Picture this...Betty has Huntington's Disease that has progressed far enough to require her to use a whelechair. Based on the charges, two counts of exploitation of the elderly, we know Betty and her husband are probably senior citizens. Graham states that out of the goodness of her heart she was helping Betty buy things such as lingerie and leather mini-skirts. Supposedly, Betty would "stand" outside of the store and point to what she wanted and Graham would make the purchase. Of course there is the little detail of the store clerks saying that Graham mentioned she was buying them for herself.
Graham says the couple isn't in as bad of shape as they claim. Others wonder that if that was the case...why did she need to sign the credit slips for them?
Sound's like Queen Graham should have plead guilty.
Posted by Dave at 10:05 PM | Comments (0) | TrackBack
April 21, 2004
Goofy SCID Press Release
SCID (Severe Combined Immunodeficiency Disease) is a serious disease and Duke University Medical Center has issue a press release touting their treatment success. They are also trying to raise awareness for this disease.
However, this medical organization says that this disease that strikes 1 in 100,000-500,000 individuals is more common than Huntington's Disease.
Reality check - Huntington's Disease strikes 1 in 10,000-30,000 individuals. Some say it's more frequent than that. Here's the press release:
Newborn Testing for Immune Disorders Could Save Lives
DURHAM, N.C., April 21 (AScribe Newswire) -- A simple, inexpensive blood test performed at birth to screen for immune disorders could dramatically increase the chance of survival for babies born with such potentially fatal disorders as severe combined immunodeficiency disease (SCID).
Physicians at Duke University Medical Center have performed stem cell transplants in 136 infants with SCID in the past 22 years. The survival rate for 38 infants receiving transplants in the first 3.5 months of life is 97 percent, but the rate drops to 69 percent for infants who were transplanted after that age, Rebecca Buckley, M.D., reports in the April 23, 2004, Annual Review of Immunology.
The main cause for the drop in survival rate is serious infections SCID babies develop in the first few months of life. Infants with SCID have little or no immune system. Without treatment, they die of infection before their first or second birthdays. But for infants without a known family history of SCID, the average age of referral for immune testing is approximately 6 months, Buckley said. "The tragedy is that most patients are critically ill by then," she said.
Buckley believes that all newborns should be screened for immune deficiency disorders at birth. "SCID is a pediatric emergency. There is no screening for any primary immunodeficiency disease at birth or during childhood and adulthood in any country. Thus, most patients are not diagnosed until they develop a serious infection, which certainly adversely affects the outcome of therapy," said Buckley, a professor in Duke's division of pediatric allergy and immunology.
Early treatment also reduces costs -- a transplant in the first three months of life can cost less than $50,000, but the cost of care skyrockets up to millions of dollars for seriously ill patients, with less guarantee of success. And SCID patients who received stem cell transplants from related donors within the first 28 days of life developed a more robust immune system, with higher levels of T cell reconstitution and output from the thymus gland. T cells are white blood cells that are essential for normal function of the immune system, Buckley reports.
Nearly all SCID cases can be diagnosed at birth by counting the number of lymphocytes, a type of white blood cell, present in umbilical cord blood, Buckley said. Infants with SCID have a profound deficiency of lymphocytes, due to the deficiency of T cells that help fight infections. Children with other immune disorders could also be identified through this test, which costs an average of $50 at a commercial laboratory. Researchers at the National Humane Genome Research Institute are developing a test for immunodeficiency disorders that could be performed on the small blood sample now taken from newborns to screen for certain metabolic disorders.
Nine forms of SCID have been identified in the past 10 years, caused by mutations of single genes. However, Buckley has treated 30 patients without mutations in the known SCID genes, making it likely other causes are yet to be discovered. The most common form of SCID is X-linked recessive, a mutation inherited on the X chromosome. Because X-linked recessive genes are expressed in girls only if a child receives two copies of the gene -- one from each parent -- the disease is more common in boys, who only need one copy for an X-linked recessive gene to be expressed. SCID-X1 accounts for 46 percent of U.S. cases.
The incidence of SCID has been projected to range from one in every 100,000 to 500,000 births -- more frequent than disorders such as Huntington's disease. "However, no one truly knows how common this disease is. I suspect that it is much more common than thought because a lot of SCID patients probably die before their disease is recognized," Buckley said.
Buckley and her colleagues at Duke University Medical Center treat SCID patients via stem cell transplants derived from donor bone marrow, typically from a parent or matched sibling. Transplant recipients do not need pretransplant chemotherapy or prophylactic treatment for graft-versus-host disease. Infants with SCID have a complete absence of T cell function, so they cannot reject the transplants. The bone marrow is processed to remove T cells, preventing the donor T cells from attacking the recipient, known as graft-versus-host disease. Mature, donor-derived, T cells typically appear in SCID patients within 90 to 120 days after transplant. The success of treatment varies among different forms of SCID.
Clinicians are striving to improve the success of transplant therapy and create more robust immune systems by giving higher numbers of stem cells in preparations nearly devoid of T cells, Buckley added. "If the imperfect results seen with stem cell therapy in the past were due to an insufficient number of stem cells, this approach should result in better immune reconstitution. The only remaining obstacle would then be to ensure diagnosis is made early before untreatable infections develop," she said.
Of the 136 SCID patients treated at Duke, 105 (77 percent) are alive. None show any evidence of susceptibility to opportunistic infections and most are in good general health. The oldest is 22 years of age. All 15 recipients of marrow from perfectly matched donors and 89 of the 121 recipients of T cell-depleted marrow from related donors are among the survivors.
Of the 38 infants transplanted during the first 3.5 months of life, 37 (97 percent) survive, compared to 68 survivors among the 98 transplanted after that age (69 percent success). Twenty-four of the 31 deaths occurred from viral infections. Graft-versus-host-disease (GVHD) occurred in 40 of the 121 patients given T cell-depleted parental bone marrow, but most of the GVHD was mild and required no treatment; there were no deaths from GVHD. In 35 of 40 GVHD cases, the complication occurred when there was persistence of transplacentally transferred maternal T cells.
Posted by Dave at 06:52 PM | Comments (0) | TrackBack
April 20, 2004
Massacree HD
From the Hunt-Dis List - a very worthwhile fundraiser:
This year's Garbage Trail Walk to Massacree HD is Sunday, May 16th in Great Barrington and Stockbridge, Massachusetts. The Garbage Trail is a trail of the key sites in The Alice's Restaurant Saga: "The Church," now the Guthrie Center, the old "Closed on Thanksgivin'" Stockbridge Dump, the Stockbridge Police (Officer) Station and Theresa's Stockbridge Café, site of the original Alice's Restaurant. It's a 6.6 mile pledge walk that starts at the Center and ends at Theresa's. Volunteers with VW Microbusses will shuttle walkers back to The Church in their vans. Water stops will be serving ice cream cones and other healthy snacks!
Last year we raised over $20,000. We gave half to Casa Hogar in Venezuela through the Hereditary Diseaase Foundation and the other half to the Marjorie Guthrie Fund of the International Huntington's Association...
And if you'd like to send us a contribution, please send it to:
Jim Pollard
63 Wentworth Avenue
Lowell, MA 01852.
Please make the check payable to "HD Walk/Guthrie Center."
Posted by Dave at 06:30 PM | Comments (2) | TrackBack
April 19, 2004
CoQ10 On The Fast Track
In a sparsely worded press release Tishcon Corporation announced that they have received orphan drug status for CoQ10. This speeds up the time when CoQ10 might be an approved drug for Huntington's Disease. For those with insurance that could save them $100+/mo in costs over what is paid today.
Here's the press release:
Tishcon Corp. Receives Orphan Drug Designations for Coenzyme Q10 in the Treatment of Pediatric Congestive Heart Failure and Huntington's Disease
WESTBURY, N.Y., April 19 /PRNewswire/ -- Tishcon Corp. announced that the
U.S. FDA has granted the firm Orphan Drug Designations for UBIQUINOL (Coenzyme
Q10) in the treatment of
1- Pediatric Congestive Heart Failure, and
2- Huntington's Disease
Tishcon Corp. was also granted an Orphan Drug Designation for Coenzyme Q10
(Ubi-Q-Gel(R)) in the treatment of Mitochondrial Cytopathies, in 1999.
Posted by Dave at 08:40 PM | Comments (0) | TrackBack
April 18, 2004
Day Off
I'm taking a day off today. No postings todays.
Back again tomorrow.
Posted by Dave at 10:46 PM | Comments (0) | TrackBack
April 17, 2004
Antidepressants Dangerous?
Keep an eye out on the debate about antidepressants over the next year.
There are a number of legislators and medication luddites who fail to understand "cause and effect". Their argument is this...
'Johnny was on an antidepressant, Johnny committed suicide, therefore...the antidepressant caused suicide.'
This is a bunch of bull. Antidepressants are taken for 'depression', hence the name. A major symptom of depression is suicide. The only thing that the 'Johnny' example proves is the antidepressant didn't do it's job.
Where treating depression gets difficult is that there is no one antidepressant that works for everybody. So getting someone on the right medication can be a challenge for doctors. Especially if the patient isn't communicating effectively about the status of their symptoms. Some people aren't on the right medication and are at risk of all the symptoms of depression.
The true test for an antidepressant is to measure the number of suicides among users and compare that number against the number of suicides committed by equally depressed people who are not on a medication.
Now that the politicians are involved, watch out for the potential of stupid policies being enacted.
If you're on an antidepressant and it is helping, stay on it. If you don't think it is helping, talk with your doctor BEFORE discontinuing!
Posted by Dave at 11:13 PM | Comments (0) | TrackBack
April 16, 2004
Finding More HD Genes
Not HD exactly, but genes that modify the progression of Huntington's Disease.
Several weeks ago a HUGE study was released (and mentioned here) based on the Venezuelan research of Dr. Nancy Wexler and many other researchers. The upshot of the study was that factors outside of the HD gene accounted for 40% of variability in the age of onset.
This doesn't just refer to evironmental factors, this also refers to 'modifying genes' and researchers are now starting to look at genes that might be utilized to delay the onset of Huntington's. (Another article here.)
Fortunately, with all the advances in the last few years, this research is much faster and cheaper to accomplish than it was back in 1993 when the HD gene was found.
Posted by Dave at 11:46 AM | Comments (1) | TrackBack
April 15, 2004
HDSA Nominations
Another bit from the Hunt-Dis list:
HDSA is soliciting nominations for awards to presented at the annual convention in June. Nominations need to be received by May 15th.
Information and the nomination form are available here.
Posted by Dave at 11:59 PM | Comments (0) | TrackBack
April 14, 2004
A Useful Analysis
There's an interesting article from two researchers in the International Clinical Psychopharmacology journal.
They've taken a look at the current research to discuss what what treatments are useful to those with Huntington's Disease. Here's what they recommend:
For movement disorders:riluzole, olanzapine & amantadine
For depression and various psychiatric issues:
selective serotonin reuptake inhibitors & mirtazapine
For 'optimal managment':
adjuvant psychotherapy, physiotherapy & speech therapy
The also discuss the possible usefulness of:
minocycline, unsaturated fatty acids & riluzole
Here's the abstract:
Int Clin Psychopharmacol. 2004 Mar;19(2):51-62.
Huntington's disease: present treatments and future therapeutic modalities.
Bonelli RM, Wenning GK, Kapfhammer HP.
University Clinic of Psychiatry, Karl-Franzens University Graz, Graz; University Clinic of Neurology, University of Innsbruck, Innsbruck, Austria. raphael.bonelli@klinikum-graz.at
Huntington's disease (HD) is a devastating neuropsychiatric disorder for which therapeutic interventions have been rather fruitless to date, except in a slight symptomatic relief. Even the discovery of the gene related to HD in 1993 has not effectively advanced treatments. This article is essentially a review of available double-blind, placebo-controlled trials of therapy for this condition which also includes relevant open label trials. Unfortunately, HD research has tended to concentrate on the motor aspects of the disorder, whereas the major problems are behavioural (e.g. dementia, depression, psychosis), and the chorea is often least relevant in terms of management. We conclude that there is definitely poor evidence in management of HD. The analysis of the 24 best studies fails to result in a treatment recommendation of clinical relevance. Based on data of open-label studies, or even case reports, we recommend riluzole, olanzapine and amantadine for the treatment of the movement disorders associated with HD, selective serotonin reuptake inhibitors and mirtazapine for the treatment of depression, and atypical antipsychotic drugs for HD psychosis and behavioural problems. Moreover, adjuvant psychotherapy, physiotherapy and speech therapy should be applied to supply the optimal management. Finally, some cellular mechanisms are discussed in this paper because they are essential for future neuroprotective modalities, such as minocycline, unsaturated fatty acids or riluzole.
PMID: 15076012 [PubMed - in process]
Posted by Dave at 07:34 PM | Comments (0) | TrackBack
April 13, 2004
Genetic Discrimination Interview
The American Medical News has published an interview with Dr. Francis Collins, head of the Human Genome Research Institute, on genetic discrimination.
Here's what the good doctor has to say about genetic discrimination and testing:
Q: Would you have any qualms about undergoing genetic testing in the current environment?
A: Yes, I would. If I were in a situation where I was confident that a particular genetic test was highly indicated and where the results might give me the information that could provide me with an opportunity to reduce my risk, I would be very interested in the test, but I would have to weigh that against the likelihood that this could result in damage to my employability or to my ability to get health insurance. And that would be a serious factor. I would not take that lightly. I do think the risks, while not huge, are real, and until we have a circumstance where that is effectively outlawed by well-written federal legislation, I would be quite uneasy.
As they say...read the whole thing.
Posted by Dave at 01:18 AM | Comments (0) | TrackBack
April 12, 2004
Medicare Drug Comparisons
According to the New York Times, the government is going to start publishing the prices of medications for Medicare participants. The price comparisons will include both generic and brand-name medications. This will allow consumers to compare the benefits of various drug discount cards (coming in a couple of months).
Keep your eye on www.medicare.gov for more information.
Posted by Dave at 10:08 PM | Comments (0) | TrackBack
April 11, 2004
HD In Comic Books?
Well...not so much in the comic books but in a 'graphic novel'.
Michael Berry from the San Francisco Chronicle writes a review of the 128 page hard cover graphic novel titled "It's a Bird...". It tells the story of a man who has just gotten a his dream job, doing the Superman comic books. However it comes at a time when his personal life isn't such a dream...including the spectre of Huntington's Disease in his family.
This is another unexpected way HD is being introduced to people who otherwise never would have heard of the disease. This sounds like a very interesting read. Here's what the reviewer had to say:
Seagle and Kristiansen's "It's a Bird ..." takes a far more subtle approach to its fantastic underpinnings. As a writer of comic books, Steve has just received a chance at one of the industry's most coveted gigs, chronicling the adventures of Superman. He isn't sure he wants it, though. He's not getting along with his girlfriend, his father has gone missing and his mother is at her wit's end with worry. Worse, the specter of a debilitating illness - - Huntington's disease -- hangs over Steve and his family. How can he write about the Man of Steel when he's so frightened by the notion that his own genes might betray him?
Without allowing the project to become maudlin, Seagle masterfully mixes elements of autobiography with one of the great mythological constructs of 20th century America. Kristiansen's painting, with its palette of browns, grays and greens, strikes just the right notes of the commonplace and the majestic. "It's a Bird ..." does something unique with the graphic novel, and it's well worth the time of any reader, even if she or he has no real interest in the true identity of Clark Kent.
Posted by Dave at 10:07 AM | Comments (0) | TrackBack
April 10, 2004
Marie and Bike For A Cure
Read this article on Marie Nemec, written several years ago, on her riding her bike to raise money for the Huntington's Disease community.
She's still going at it and with great success! Please read these earlier articles (Here and Here) and see if there isn't some way you can help. This year she is riding from Albuquerque to St. Louis in the "Kick HD on Route 66" tour!!!
This is for a great cause and she is a great lady so please help in any way you can.
Posted by Dave at 08:29 AM | Comments (0) | TrackBack
April 09, 2004
Controlling Health Care
MedPundit points to an interesting article by Jane Galt called "Who Controls Our Health Care".
At least it will be interesting to policy wonks. The general argument that is being made is that the government already controls a substantial portion of the nation's health care. There is also some interesting (and occasionally intelligent) discussion in the following comment section. There is also a lot of mythology about the quality of the Canadian health care system.
Posted by Dave at 11:08 PM | Comments (0) | TrackBack
April 08, 2004
USF Study For Chorea Medication
This was posted on the Hunt-Dis list. If you live in the Tamba Bay area, you may wish to check this out.
Here's the information:
Dr. Zesiewicz at the University of South Florida is conducting a study involving Huntington's Disease and its symptoms. We are looking for persons diagnosed with Huntington's disease who are interested in helping us understand the effect of an experimental medication in controlling involuntary movements called Chorea. We are recruiting for this study for the entire month of April. This study is expected to last about 7 weeks. If you are interested in getting involved and/or have questions regarding this research, please call our office at (813) 974-5909 or email us at: jmaldona at hsc.usf.edu
Best Regards,
John Maldonado, B.S.
USF Dept. of Neurology
Division of Movement Disorders
Here is some Q & A that you might also want to know about this...
1. What is the experimental medicine? Keppra
2. Is this study for any person with HD experiencing Chorea in the TampaBay and surrounding area or is it open to anyone from Florida or just the TampaBay area. Anyone in Florida who can get to USF 5 times thorough out the 7 weeks.
3. Is this study part of the Huntington Study Group [HSG] clinical
trials? No it's a separate trial through the dept of neurology
4. Can they not be taking any existing medication for chorea? If so,
what? Yes they can be taking other medication, however there are some that are certain limitations which would be gone over the first visit.
5. Will there be any cost to the person for participating in this
clinical trial? No cost or compensations. They will not be charged for study visits or medication
6. Any other exclusions why a person would not be accepted for this
clinical study? Not at this time.
7. Is Dr. Zesiewicz associated with Dr. Sanchez-Ramos or Dr. Hauser or another doctor? She is actually associated with both. She works with Dr. Sanchez with Huntington's disease patients from time to time.
Posted by Dave at 02:39 AM | Comments (0) | TrackBack
April 07, 2004
DNA Research Getting Cheaper
FuturePundit has an excellent piece on the declining costs of DNA sequencing.
Simply put...lower costs means that there will be more genetic research done. In just two years the cost of mapping DNA Single Nucleotide Polymorphisms has dropped from 50 cents to 1 cent per. That's a real cost savings when you need to do 100,000 of them!
For genetic diseases such as Huntington's, this is just the kind of news we need to hear.
Posted by Dave at 09:49 PM | Comments (0) | TrackBack
April 06, 2004
Free Prescription Medicines
Pharmaceutical companies give out over $3 Billion worth of medications every year to those who can't afford them.
Now there is a website where you can get more information: www.helpingpatients.org
Here's the press release:
The Best Deal on the Internet: Safe and Free Prescription Medicines
Tuesday April 6, 12:43 pm ET
Web Site Can Serve as 'One-Stop-Shop' for Many Patient Assistance Programs
Pharmaceutical Company-Sponsored Programs Give More Than $3 Billion Annually to Needy Patients
WASHINGTON, April 6 -- Millions of Americans have turned to the Internet for more affordable drug prices, but they may be surprised to learn that for millions of consumers the best deals are available from pharmaceutical companies in the form of patient assistance programs.
In a continuing effort to help doctors and an estimated 6.2 million low- income patients take advantage of free and significantly discounted drugs, America's pharmaceutical companies are launching a Web-based tool on www.helpingpatients.org that provides easier and faster access to more than 400 different medications through the 40 different patient assistance programs offered by the pharmaceutical companies that manufacture those medications. Patient assistance programs have been supported by the industry for many years and continue to grow, but this site gives patients and health care providers "one-stop-shopping" for the various programs.
The site's new feature, "Fast Access," allows patients or their healthcare provider to enter data once and that data is then fed to the various assistance programs to determine where there might be a match for that patient. All applications must be approved by the patient's health care provider, so patients may use the tool, print out the forms and then work with their health care provider to submit the applications or the health care provider may handle the entire process for the patient.
"Many of the people served by our members can benefit from this program," said a spokesperson of the National Medical Association, the nation's largest association of African-American physicians. "This is a great tool to help ensure that all of our patients get safe and effective medications."
The site hosted by the Pharmaceutical Research and Manufacturers of America (PhRMA) also includes an online database of patient assistance programs that provide help for millions of people who lack prescription drug coverage. In addition to the programs mentioned above, the database created and funded by the pharmaceutical industry also provides patients with information regarding 1,000 more medicines offered through over 100 other industry-sponsored programs and 185 government and privately-sponsored patient assistance programs.
"We talked with patients and doctors alike about how we can make the process of applying to patient assistance programs simpler, faster and easier," announced Miles White, PhRMA's chairman of the board of directors and chairman and chief executive officer of Abbott Laboratories, at PhRMA's Annual Meeting on April 3, 2004. "We listened and now we are acting on what we have learned."
In 2003, patient assistance programs helped more than 6.2 million underinsured or uninsured patients obtain nearly 18 million prescriptions. The estimated wholesale value of medicines distributed though company patient assistance programs last year totaled almost $3.4 billion.
"We know that many people have difficulty getting the medicines they need; this is one important piece of an answer," said PhRMA President and CEO Alan F. Holmer.
Medicines available through the program include many of the newest and most innovative brand-name treatments available, including medicines that treat:
* Cancer
* High Cholesterol
* Diabetes
* High blood pressure
* Stroke
* Depression
* Schizophrenia
* Alzheimer's
Consumers can access these programs and download a free copy of the patient assistance program directory at www.helpingpatients.org, or at PhRMA's Web site www.phrma.org. PhRMA also publishes a printed version of the Directory of Prescription Drug Patient Assistance Programs, which lists programs, the companies that offer them, available medicines, basic eligibility requirement and contact persons.
The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country's leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives. PhRMA members invested an estimated $33.2 billion in 2003 in discovering and developing new medicines. PhRMA companies are leading the way in the search for new cures.
PhRMA Internet Address: www.phrma.org
Posted by Dave at 07:45 PM | Comments (0) | TrackBack
April 05, 2004
Another HD Hero
Bill Jaffe is raising money to save the children of a man with Huntington's Disease. He will be running a half-marathon as part of his efforts to raise $50,000 for HD research. You can read about it here in the Richmond News.
Posted by Dave at 11:13 PM | Comments (0) | TrackBack
April 04, 2004
What About An HD Vaccine?
Here's an interesting article from HealthDay on vaccines for cancer.
What does that have to do with Huntington's Disease? ...Possibly everything.
Scientists are now developing vaccines that 'teach' the body to attack certain proteins and enzymes. It is entirely possible that scientists could develop a vaccine that targets the 'bad' huntingtin protein while leaving the good one alone.
There is work going on for a Huntington's vaccine and there is a lot of research going on for cancer vaccines. Success in one area helps the other. Here's the article.
Posted by Dave at 06:36 PM | Comments (0) | TrackBack
April 03, 2004
What A Wonderful Week!
Every once in a while we are fortunate to have a great week. This was one of those weeks.
In the past week:
1.) A new study finds that Paxil protects brain cells in HD mice.
2.) The protein that makes the huntingtin protein toxic was announced.
3.) Several successful Hoop-a-thons were conducted.
4.) We saw a person with HD memorialized in bronze for firefighters.
5.) We heard about a movie being produced on Huntington's Disease.
6.) The Australian HD organization got a $400,000 gift.
and my favorite...
7.) A wonderful lady wrote to say that she and a friend have found that there is life after a positive diagnosis for HD.
There is little better than to hear that someone will not let a 'diagnosis' rob them of their lives, their families, and their friends.
Posted by Dave at 08:11 PM | Comments (1) | TrackBack
April 02, 2004
HD Firefighter Memorialized
Redmond Washington dedicated a memorial yesterday for Redmond Firefighters. The bronze sculpture shows Bert Eymberts on his knee offering his hand.
Bert died two years ago from Huntington's Disease. His wife Robin Eymberts was there for the dedication. Seattle Times photographer has a wonderful picture of Robin holding the outstretched hand.
Bert was a firefighter for over 20 years. One more example of how those with HD have helped others.
Posted by Dave at 11:57 PM | Comments (0) | TrackBack
April 01, 2004
Have They Found A Simple Cure?
UC Irvine has come out with more great news in the fight against Huntington's Disease.
They found a protein, SUMO-1, that is responsible for making the mutated huntingtin protein toxic to the brain cell. No SUMO, no early cell death!
So what does this mean for a cure? Well, it means that a relatively not-difficult-to-develop drug could block the SUMO-1 protein. This COULD result in a very good treatment for Huntington's Disease.
So here's the caveat: It's possible that SUMO-1 has some needed function in the body and that blocking it will cause other unwanted problems. Another issue could be the difficulty in developing a drug that can readily cross the blood-brain barrier.
Assuming for a moment that a relatively effective treatment can be developed quickly, it will be six or seven years before it becomes approved for humans. Most potential drugs never make it that far.
No matter what, this is yet ANOTHER promising treatment path that researchers have identified. Many will not pan out but I do believe one will, perhaps even before the decade is out. There is hope!
Here's UC Irvine's press release:
Identification of protein trigger may lead to treatment for Huntington's disease
Irvine, Calif., April 1, 2004
UC Irvine scientists have identified a protein they believe can trigger the onset of Huntington’s disease, an incurable, fatal genetic brain disorder that affects some 30,000 people in the United States.
In studies on fruit flies and cultured cells, the researchers found that a small protein called SUMO-1 modifies the mutated protein linked to the disease, changing its chemical properties and making it significantly more toxic. The finding suggests that drugs developed to block SUMO-1 from modifying the Huntington’s disease protein may be effective in delaying disease progression. Study results appear in the April 2 issue of Science.
Lawrence Marsh of the School of Biological Sciences, Leslie Thompson and Joan Steffan of the College of Medicine, and colleagues studied how SUMO-1 interacts with the Huntington’s disease protein, Htt.
The role of the small SUMO protein in a cell is to modify the activities of larger proteins, such as Htt. The researchers found that in modifying the Htt protein, SUMO-1 initiates changes that make the Htt protein more toxic. Eventually, during the disease state, the toxic Htt protein disrupts the normal functions that are essential to brain cell health, which lead ultimately to cell death.
“We also found that blocking SUMO-1 from binding to the Htt protein prevented Htt from becoming so toxic,” Marsh said. “SUMO-1 seems to play an important role in disease progression, and this study points to new avenues of therapy that bring us closer to being able to target the cause rather than the symptoms of this devastating disease.”
Since Huntington’s is a dominant disease, a child with one parent who carries the gene that creates these mutated proteins runs a 50 percent chance of getting Huntington’s disease. The disorder is progressive, and while typically a late-onset disease, symptoms can appear in childhood. It causes uncontrolled movements, loss of intellectual capacity and severe emotional disturbances. It eventually results in death. It has been described in medical literature under a host of different names since the Middle Ages.
Most notably, the American folk singer and composer, Woody Guthrie, died in 1967 after suffering from the disease for 13 years.
In a previous study, the UCI researchers found that drugs called HDAC inhibitors, which were developed for cancer chemotherapy, were able to prevent neuron damage in fruit flies carrying mutated Htt proteins. Currently, they are studying the link between these HDAC inhibitors and SUMO-1.
Namita Agrawal, Judit Pallos, Erica Rockabrand, Natalia Slepko, Katalin Illes, Tamas Lukacsovich and Ya-Zhen Zhu of UCI; Lloyd Trotman and Pier Paolo Pandolfi of the Sloan-Kettering Institute in New York; and Elena Cattaneo of the University of Milano, Italy, assisted with the study. The Hereditary Disease Foundation, the Huntington’s Disease Society of America, the Human Frontiers Science Program and the National Institutes of Health supported the research.
About the University of California, Irvine: The University of California, Irvine is a top-ranked public university dedicated to research, scholarship and community. Founded in 1965, UCI is among the fastest-growing University of California campuses, with more than 23,000 undergraduate and graduate students and about 1,300 faculty members. The third-largest employer in dynamic Orange County, UCI contributes an annual economic impact of $3 billion.
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